Rare Disease Day 2026

Accelerating a Cure for NF2

Rare Disease Day is a global call to action for conditions that are too often overlooked. We are steadily moving toward clinical trials for NF2, and this year, we need your help to begin manufacturing the clinical-grade AAV9 (Adeno-Associated Virus 9) NF2 gene therapy product.

We are committed to full transparency about how your donations are used: The first step in the AAV manufacturing process is creating the DNA blueprint, called the plasmid. A plasmid is a small circular piece of DNA that acts as the instruction sheet to tell the gene therapy AAV9 what DNA to deliver into cells. In our case, we will deliver a healthy copy of the NF2 into the tumor.

Our Goal

We aim to raise $300,000 by February 28, 2026, to launch this first manufacturing stage. We currently need an additional $30,000. 

Thanks to the incredible generosity of our community, our Giving Tuesday campaign raised $65,000.

Our board members and ambassadors have pledged $110,000, providing strong early momentum.

An additional $5,000 has come from dedicated NF2 families.

Most recently, a $90,000 gift from the Kantor family has brought our total to $270,000.

We need your help to raise the remaining $30,000 to reach our goal.

How You Can Help

Your gift directly supports the completion of plasmid production and allows us to secure a contract and production slot for the clinical-grade viral vector. Once AAV9.NF2 manufacturing is complete, we will advance to a toxicology (safety) study and then to clinical trials.

Every contribution moves this work forward. Whether you give $25, $100, or more, you are helping accelerate progress toward a cure.

Together, we can turn hope into action.